patient
Cell-autonomous dysregulation of interferon signaling drives clonal expansion of SRSF2-mutant MDS stem/progenitor cells
Key Points
SRSF2 mutations blunt responsiveness of MDS cells to IFN stimulation and promote their clonal fitness by downregulating STAT1 expression
Proteasome inhibition restores STAT1 protein level and sensitivity of SRSF2-mutant MDS cells to IFN, suggesting a new therapeutic strategy
Natural killer cells’ functional impairment drives the immune escape of pre-malignant clones in early-stage myelodysplastic syndromes
Abstract
2025 Boston Patient and Family Conference
Event Date:
Sat, 09/27/2025 - 7:30am (EDT)
Bone Marrow Disease(s):
Conference Event Type:
2025 Chapel Hill Patient and Family Conference
Event Date:
Sat, 10/18/2025 - 7:30am (EDT)
Bone Marrow Disease(s):
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March for Marrow: Walk for PNH NJ / NY
Join us in Verona Park, NJ on Sunday, September 28, 2025 for a day of remembrance and awareness in celebration of the 19th Annual Walk for PNH.
The Walk for PNH is the nation's largest event to raise awareness and funds for PNH education and research. This inspiring event calls on participants of all ages and abilities to join in the fight against PNH.
Event Date:
Sun, 09/28/2025 - 10:30am (EDT)
Bone Marrow Disease(s):
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2025 Golf Tournament in Memory of Kimberly Owens
As we prepare for the upcoming golf tournament, benefiting AAMDSIF a nonprofit organization that helps people living with PNH, it is with a heart full of fond memories that we take a moment to reflect on Kim, whose spirit truly embodied kindness and positivity. She always had a smile on her face, uplifting everyone around her, even on challenging days.
Event Date:
Thu, 05/29/2025 - 8:00am (EDT)
Bone Marrow Disease(s):
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Hack for Marrow
Hack for Marrow is a week-long global hackathon designed for teenagers aged 13 to 19. Whether you're a passionate coder or exploring programming for the first time, we’d love to have you join us!
Participants may compete individually or in teams of up to three members. To register, simply click the “Register” button above to sign up as an individual or with your team.
Event Date:
Fri, 08/01/2025 - 12:00am (EDT) to Fri, 08/08/2025 - 11:59pm (EDT)
Bone Marrow Disease(s):
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AAMDSIF Joins Coalition Urging the Administration to Preserve our Nation’s Newborn Screening Program
A coalition of 272 organizations signed a public letter to the Secretary, Department of Health and Human Services, addressing the decision to eliminate the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). While each state may add on additional screenings, this Committee ensured standardized measurements and procedures, to endure early detection of newborn conditions.
AAMDSIF signed the letter, attached below, to endorse maintaining the Advisory Committee on Heritable Disorders in Newborns and Children.
Judy's Story of PNH - Always Moving Forward - 2025 Kansas City Patient and Family Conference
Judy talks about her PNH journey from diagnosis, treatment decisions and moving forward.